OCTAPHARMA

Octapharma announced today that pre-clinical data for SubQ-8 were
presented during a symposium at the recent World Federation of
Hemophilia (WFH) 2018 World Congress in Glasgow, UK. SubQ-8, currently
under development, is a recombinant FVIII from a human cell line for
subcutaneous administration. SubQ-8 is based on Octapharma’s human cell
line-derived rFVIII product simoctocog alpha combined with a fragment of
the von Willebrand Factor (VWF) protein, and harnesses the protective
power of VWF in an innovative approach to facilitate transportation of
FVIII into the circulation.

Regular intravenous administration of FVIII poses a considerable burden
to people with haemophilia A and their families. Alternative routes of
administration may help to alleviate some of this burden and improve
adherence to prophylaxis therapy.

This symposium, entitled ‘Taking FVIII into the future: The
development of subcutaneous recombinant human FVIII for the treatment of
haemophilia A’
, reviewed the challenges that intravenous
administration poses to people with haemophilia A on a daily basis, and
presented data on the uptake of SubQ-8 into the vasculature following
subcutaneous administration in animal models.

David Lillicrap (Queens University, Ontario, Canada) chaired the
symposium and introduced the relevance of SubQ-8 within the rapidly
advancing haemophilia treatment field. Cedric Hermans (Saint-Luc
University Hospital, Brussels, Belgium) discussed the important role of
FVIII, and the use of FVIII therapy as a natural approach to restoring
blood clotting in haemophilia A. The proven benefits of FVIII
prophylaxis include protecting from devastating intracranial bleeds and
reducing the risks of long term joint damage. Dr Hermans also presented
the challenges and burdens of intravenous infusion of FVIII, which may
present a hurdle to initiating and adhering to prophylaxis. As a result,
there is demand for a simpler FVIII administration method.

Christoph Kannicht (Octapharma Research & Development, Berlin, Germany)
explained the rationale for the development of SubQ-8, and addressed the
challenge of achieving sufficient FVIII bioavailability after
subcutaneous infusion. Pre-clinical data in the animal models showed
45.5% bioavailability of FVIII after subcutaneous administration of
SubQ-8, and a 3.6-fold prolongation of FVIII half-life compared with
FVIII intravenous infusion. Andreas Tiede (Hannover Medical School,
Hannover, Germany) addressed the immunogenicity risk of protein
therapeutics administered subcutaneously or intravenously, concluding
that there is no evidence for a difference in risk. The immunogenicity
of SubQ-8 has been studied in mice and subcutaneous administration of
SubQ-8 resulted in a slower development of anti-FVIII antibodies than
intravenous administration of FVIII.

The data presented represent a promising basis for future clinical
studies of SubQ-8. Larisa Belyanskaya, Head of Octapharma’s IBU
Haematology, said “WFH was a fantastic platform to share the most
recent data on the development of our new subcutaneous FVIII product,
SubQ-8. We at Octapharma believe that SubQ-8 could play an important
role in ensuring patients receive optimal treatment for haemophilia A
”.
Olaf Walter, Board Member of Octapharma, added that “Octapharma is
committed to improving patients’ lives and we are proud to be developing
a product with such potential for the haemophilia field. Presenting our
preclinical data at WFH is an important step in realising our goal
”.

About SubQ-8

SubQ-8 is Octapharma’s developmental rFVIII product for subcutaneous
administration, currently in the preclinical stage.

About Haemophilia A

Haemophilia A is an X-linked hereditary disorder caused by FVIII
deficiency which, if left untreated, leads to haemorrhages in muscles
and joints and consequently to arthropathy and severe morbidity. FVIII
replacement prophylactic treatment reduces the number of bleeding
episodes and the risk of permanent joint damage. This disorder affects
one in every 5,000 to 10,000 men worldwide. Globally, 75% of haemophilia
cases are left undiagnosed or untreated. The development of neutralising
FVIII antibodies (FVIII inhibitors) against infused FVIII represents the
most serious treatment complication. The cumulative risk of FVIII
inhibitor development is reported to be currently up to 39%.

About Octapharma

The vision of Octapharma is “Our passion drives us to provide new health
solutions advancing human life”. Headquartered in Lachen, Switzerland,
Octapharma is one of the largest human protein manufacturers in the
world, developing and producing human proteins from human plasma and
human cell lines. As a family-owned company, Octapharma believes in
investing to make a difference in people’s lives and has been doing so
since 1983; because it’s in our blood. Our company values are Ownership,
Integrity, Leadership, Sustainability and Entrepreneurship.

In 2017, the Group achieved €1.72 billion in revenue, an operating
income of €349 million and invested €287 million to ensure future
prosperity. Octapharma employs around 7,700 people worldwide to support
the treatment of patients in 113 countries with products across three
therapeutic areas: